The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases
caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting
various tissues throughout the body based on the specific enzyme deficiency. These
disorders are characterized by their progressive nature and a variety of somatic
manifestations and neurological symptoms. There are established treatments for some MPS
disorders, but these mostly alleviate somatic and non-neurological symptoms and do not …